Your views matter about bone health. There’s still time to take part in an important survey to improve bone health in Duchenne. The links below will …
ReveraGen receives FDA fast track designation for Vamorolone
ReveraGen has announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for vamorolone (VBP15) for the treatment …
ReveraGen receives FDA fast track designation for VamoroloneRead More
Translarna Managed Access Agreement frequently asked questions
We recently emailed you to update you on the Managed Access Agreement for Translarna. We know that some of you had questions about this. To try and …
Translarna Managed Access Agreement frequently asked questionsRead More
Papworth Trust’s Disability in the UK: Facts and Figures 2018
Did you realise that disabled people are TWICE as likely to be unemployed than non-disabled people? Are you aware that there are currently 1.8 …
Papworth Trust’s Disability in the UK: Facts and Figures 2018Read More
Ethics and Social Science – a community in partnership
Last month the Ethics and Social Science Genomics England Clinical Interpretation Partnership (GeCIP) held its first national community meeting in …
Ethics and Social Science – a community in partnershipRead More
Wave Life Sciences financial results and business update
Wave Life Sciences have reported financial results for the fourth quarter and full year ended December 31, 2017, and provided a business …
Wave Life Sciences financial results and business updateRead More
You have an important voice – make it heard in the discussion about Raxone and the EAMS
What is the EAMS? The Early Access to Medicines Scheme (EAMS) aims to give patients with life threatening or seriously debilitating conditions, access …
You have an important voice – make it heard in the discussion about Raxone and the EAMSRead More
Summit opens enrolment for people who have taken part in previous Ezutromid trials
Summit Expands Enrolment in PhaseOut DMD Phase 2 Clinical Trial to Include Planned Additional Group …
Summit opens enrolment for people who have taken part in previous Ezutromid trialsRead More
Antisense receive approval for Phase II trial in Australia
Antisense Therapeutics have received approval from the Royal Children’s Hospital (RCH), Melbourne Human Research Ethics Committee, to …
Antisense receive approval for Phase II trial in AustraliaRead More
Recognising early signs of Duchenne videos featured on BBC Look North
One of our wonderful families were featured in the news this week, as 7 year old Oliver and his Mum, Alix were interviewed by BBC Look North. Watch …
Recognising early signs of Duchenne videos featured on BBC Look NorthRead More
Recognising Neuromuscular Disorders eLearning module is now live
E-learning resources support paediatricians and other child health professionals to achieve the essential competences in a number of important …
Recognising Neuromuscular Disorders eLearning module is now liveRead More
Shelley Simmonds joined Findacure on the radio to celebrate Rare Disease Day
One of our wonderful Trustees, Shelley Simmonds joined Findacure on Cambridge 105 to celebrate Rare Disease Day 2018. Listen to the whole …
Shelley Simmonds joined Findacure on the radio to celebrate Rare Disease DayRead More
